Sangamo has made another gene therapy deal to keep the lights on for a few more months, extending its funding by another three months until at least mid-2025.
On Thursday, the struggling California biotech said it would license its experimental brain-targeting adeno-associated virus capsid to Astellas for $20 million in cash, plus milestone payments, for an undisclosed neurological disease target.
The virus is designed to cross the blood-brain barrier that keeps most drugs out. It’s the second time this year Sangamo has struck an AAV deal that adds to its slim bank account and delays a possible bankruptcy.
In March, it told investors it only had enough funds to survive into the third quarter. In August, a lifeline came through
a partnership with Genentech
, which paid $50 million upfront for preclinical-stage gene therapies for Alzheimer’s and an undisclosed condition, both reliant on Sangamo’s new AAVs.
Although existing capsids like AAV9 can be injected directly into the spinal cord or brain, developing delivery vehicles that can cross into the brain from a simple blood infusion has proven difficult. Sangamo has
said
its new capsid, STAC-BBB, was 700 times better at expressing genes in the brains of monkeys compared to AAV9.
Several biotech companies, including Affinia Therapeutics, Capsida Biotherapeutics, Dyno Therapeutics, Voyager Therapeutics and others, are racing to develop brain-penetrant capsids. An Astellas spokesperson said that after an “an extensive evaluation,” the company decided Sangamo’s was “a best-in-class capsid for the treatment of serious genetic diseases” of the central nervous system.
The Genentech money was enough to sustain Sangamo until the first quarter of 2025. A Sangamo spokesperson told
Endpoints News
that with the Astellas license, “combined with other potential levers in development, Sangamo has line of sight to extending the cash runway into Q2 2025 and beyond.”
Astellas could expand its work on up to four more targets, and Sangamo could earn up to $1.3 billion in license fees and milestone payments across the five potential programs.
On Wednesday evening, Sangamo’s stock
$SGMO
was up roughly 370% since the start of 2024, buoyed by its October announcement that it plans to ask for an
accelerated approval
of its gene therapy for Fabry disease, without the need for a confirmatory study, next year. Its stock also jumped this week when Roche announced it would
halt development
of a competing gene therapy for hemophilia A. But the company’s $550 million market cap is still among the lowest in its 24 years as a public company and is down 82% since its IPO in 2000.
